A Multicenter, Multi-national Open-label Program to Provide BMN 190 to Patients Diagnosed With CLN2 Disease
- Status
- APPROVED_FOR_MARKETING
- Phase
- N/A
Clinical Trials Related to BioMarin Pharmaceutical
Related studies linked through entity extraction aliases.
Extension Study of BMN 044 in Duchenne Muscular Dystrophy (DMD)
- Status
- TERMINATED
- Phase
- PHASE2
A Study to Evaluate Long-Term Safety, Tolerability, & Efficacy of BMN 111 in Children With Achondroplasia (ACH)
- Status
- ACTIVE_NOT_RECRUITING
- Phase
- PHASE2
A Safety, Tolerability, and Efficacy Study of BMN 190 in Pediatric Patients < 18 Years of Age With CLN2 Disease
- Status
- COMPLETED
- Phase
- PHASE2
The Effectiveness of Kuvan in Amish PKU Patients
- Status
- COMPLETED
- Phase
- PHASE4
Extension Study of Drisapersen in DMD Subjects
- Status
- NO_LONGER_AVAILABLE
- Phase
- N/A
Achondroplasia Natural History Multicenter Clinical Study
- Status
- RECRUITING
- Phase
- N/A
New Imaging Technology to Assess Effect of Enzyme Replacment Therapy on Eye Disease Progession in Mucopolysacchardiosis
- Status
- UNKNOWN
- Phase
- N/A
Gene Therapy Study in Severe Haemophilia A Patients (270-201)
- Status
- COMPLETED
- Phase
- PHASE1/PHASE2
Talazoparib in Combination With Gemcitabine and Cisplatin in Patients With Advanced Solid Tumors
- Status
- WITHDRAWN
- Phase
- PHASE1
An Extension Study to Evaluate the Long-Term Efficacy and Safety of BMN 190 in Patients With CLN2 Disease
- Status
- COMPLETED
- Phase
- PHASE1/PHASE2
A Phase 3 Substudy to Evaluate Executive Function in Adults With PKU Who Are Participating in the Phase 3 Study, 165-302
- Status
- COMPLETED
- Phase
- N/A
Phase II Trial of Talazoparib in BRCA1/2 Wild-type HER2-negative Breast Cancer and Other Solid Tumors
- Status
- COMPLETED
- Phase
- PHASE2
Talazoparib Plus Irinotecan With or Without Temozolomide in Children With Refractory or Recurrent Solid Malignancies
- Status
- COMPLETED
- Phase
- PHASE1
Collection and Storage of Human Biospecimens for Research Into Rare Diseases and Medical Conditions
- Status
- UNKNOWN
- Phase
- N/A
Dynamic Respiratory Muscle Function in Late-Onset Pompe Disease
- Status
- COMPLETED
- Phase
- N/A
Rhode Island Diastolic Dysfunction - Heart Failure
- Status
- COMPLETED
- Phase
- NA
Open Label, Extension Study of PRO044 in Duchenne Muscular Dystrophy (DMD)
- Status
- TERMINATED
- Phase
- PHASE2
A Multicenter, Multinational, Observational Morquio A Registry Study (MARS)
- Status
- COMPLETED
- Phase
- N/A
A Prospective, Noninterventional, Observational Study of Late-Onset Pompe Disease
- Status
- TERMINATED
- Phase
- N/A